Selim Corbacioglu, M.D., from the University of Regensburg in Germany, and colleagues investigated whether defibrotide decreased the incidence of veno-occlusive disease in pediatric patients undergoing HSCT. Participants included 356 patients, enrolled from 28 European university hospital or academic medical centers from January 2006 to January 2009, aged younger than 18 years, who had undergone myeloablative conditioning before allogeneic or autologous HSCT. Participants were randomly assigned to receive intravenous defibrotide prophylaxis (180 patients) or not (176 patients).

The researchers found that 12 percent of patients in the treatment group had veno-occlusive disease by 30 days after HSCT, compared with 20 percent of controls (risk difference, −7.7 percent; Z test for competing risk analysis P = 0.0488; log-rank test P = 0.0507). By day 180, adverse events were seen in 87 percent of patients in the defibrotide group versus 88 percent of controls.

"Our results suggest that defibrotide can reduce rates of veno-occlusive disease and has a good safety profile in high-risk pediatric patients," the authors write.

Several authors disclosed financial relationships with pharmaceutical companies, including Gentium, which manufactures defibrotide and partially funded the study.